Back to search results

CTI-1601 for Friedreich's Ataxia

This study is testing CTI-1601, a treatment designed to deliver human frataxin, the protein that is missing in people with Friedreich's ataxia. We want to understand its long-term safety and how well it works. You might be able to join if you have Friedreich's ataxia and are between 2 and 60 years old. This study is open to people who have participated in a previous CTI-1601 study, as well as those who have not. We will be looking for any side effects and changes in heart health, like your heart rate and how well your heart pumps blood. The study is currently enrolling up to 85 participants, but the overall status is unclear.

Study design
This is an open-label study, meaning both you and the study team will know you are receiving CTI-1601. It aims to enroll 85 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Safety and heart health will be monitored for up to 24 months.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06447025

An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia

Recruiting
PHASE2Ages 2–60InterventionalTreatment
Larimar Therapeutics, Inc.
~85 participants
Updated 2026-09-03 on ClinicalTrials.gov
What's tested:CTI-1601

At a glance

Recruiting sites
6 of 8 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of subjects with treatment-emergent adverse events (TEAEs) by System Organ Class (SOC), Preferred Term (PT) and Maximum Severity
Measured over Up to 24 months
+19 more outcomes measured

Conditions

Friedreich Ataxia

Explore related trials

Friedreich Ataxia

NCT06447025

Where you'd take part

This study runs at 8 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Children's Hospital of the University of Pennsylvania (CHOP)

    Philadelphia, Pennsylvaniastudy coordinator listed

    Recruiting

  • Clinilabs Drug Development, Corp.

    Eatontown, New Jerseystudy coordinator listed

    Recruiting

  • Morsani Center for Advanced Health Care, University of South Florida Health

    Tampa, Floridastudy coordinator listed

    Recruiting

  • Uncommon Cures

    Chevy Chase, Marylandstudy coordinator listed

    Recruiting

  • University of California Los Angeles

    Los Angeles, Californiano site contact published

    Recruiting

  • University of Iowa

    Iowa City, Iowano site contact published

    Recruiting

  • Fixel Institute for Neurological Disease, University of Florida Health

    Gainesville, Floridano site contact published

    Active, not recruiting

  • Ohio State University United States

    Columbus, Ohiono site contact published

    Active, not recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Larimar Therapeutics, Inc. · STUDY_CHAIR · Larimar Therapeutics, Inc.

Who to contact

Larimar Therapeutics, Inc.
[email protected]
844-511-9056
Email the study team

Opens a ready-to-send draft in your own email app — review before sending.

Want this trial checked against your situation?

Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.

Check eligibility for this trial ~2 min · HIPAA-protected · delete anytime
Eligibility criteria

Exclusion

Subject has a HbA1c less than or equal to 7.0%.
Subject must demonstrate sufficient dexterity and visual acuity to prepare and self-administer SC injections of CTI-1601 QD or is able to identify a caregiver who will be trained and committed to prepare and administer the daily injections.
Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only one allele) for FRDA.
Subject has any condition, disease, or situation, including a cardiac condition or disease, that in the opinion of the PI, could confound the results of the study or put the subject at undue risk, making participation inadvisable.
Subject used any investigational drug (other than CTI-1601) or device within 90 days prior to Screening.
Subject requires use of amiodarone.
Subject used erythropoietin, etravirine, or gamma interferon within 90 days prior to Screening.
Subject use of biotin supplementation that exceeds 30 mcg/day, either as part of a multivitamin or as a standalone supplement, within 7 days prior to the first dose of study drug. Biotin supplementation ≤30 mcg/day is permitted if taken at a stable dose and frequency for at least 28 days prior to Screening and there is a commitment from the subject to maintain the biotin dose throughout the study (due to interference with assays).
Subject uses more than 3 grams of acetaminophen daily.
Subject receives medication that requires SC injection in the abdomen or thigh.
Subject is unable to discontinue medications that have not been at a stable dose and frequency for at least 28 days prior to Screening.
Subject has a Screening echocardiogram (ECHO) LVEF \< 45%.
Male subject has a QTcF \> 450 milliseconds or female subject has a QTcF \> 470 milliseconds on an ECG.

What this trial measures

  • Number of subjects with treatment-emergent adverse events (TEAEs) by System Organ Class (SOC), Preferred Term (PT) and Maximum SeverityUp to 24 months

    Number of subjects

  • Change from baseline in electrocardiogram (ECG) parameters including, but not limited to, HR, RR interval, PR interval, QRS duration, QT interval, and QTcF intervalUp to 24 months

    Number change in ECG parameters

  • Change from baseline in left ventricular ejection fraction (LVEF)Up to 24 months

    LVEF indicates the percentage of change in LV volume from diastole to systole that measures how well the left ventricle of the heart pumps blood.

  • Change from baseline in left ventricular end-diastolic volume (LVEDV)Up to 24 months

    LVEDV is the amount of blood, measured in milliliters (mL), in the heart's left ventricle just before the heart contracts.

  • Number of subjects with any suicidal ideation or behavior (Categories 1-10) of the Columbia Suicide Severity Rating Scale (C-SSRS)Up to 24 months

    The Columbia Suicide Severity Rating Scale (C-SSRS) is a tool used to assess the occurrence, severity, and frequency of suicidal thoughts and behaviors. A higher score on the C-SSRS generally indicate a worse outcome, as they signify a higher level of suicidal ideation or behavior.

  • Change from baseline at each collection timepoint in tissue frataxin concentrations normalized to total protein observed in buccal cells collected from cheek swabs and skin cells collected from skin punch biopsiesUp to 24 months
  • Change from baseline in motor function as assessed by 9-hole peg test (9-HPT)Up to 24 months
  • Change from baseline in motor function as assessed by the timed 25-foot walk test (T25-FW)Up to 24 months
  • Change from baseline in neurologic function as assessed by the modified Friedreich's Ataxia Rating Scale (mFARS) total scoreUp to 24 months

    The Modified Friedreich's Ataxia Rating Scale (mFARS) is a modified neurologic scale involving direct subject participation and targets specific areas impacted by Friedreich's ataxia (bulbar, upper limb, lower limb, and upright stability), with scores ranging from 0-67 points, with higher scores indicating a greater level of disability.

  • Change from baseline in neurologic function as assessed by the upright stability subscale examination of the mFARSThrough study completion, up to 24 months

    The Upright Stability Subscale is an assessment of an individual's ability to maintain balance and stability while standing upright. It has a minimum value of 0 and a maximum value of 36. A higher score indicates a better outcome, reflecting greater stability and balance abilities while standing upright.

  • Change in activities of daily living (ADLs) as assessed by the Friedreich's Ataxia Rating Scale Activities of Daily Living (FARS_ADL)Up to 24 months

    The FARS\_ADL, scored 0 to 36, is a subscale of FARS assessing a subject's ability to complete activities of daily living. A higher score indicates a greater level of disability. The FARS\_ADL questionnaire will be performed at the timepoints indicated in protocol.

  • Change from baseline in total fatigue score and all the subscale scores as assessed by the Fatigue Impact Scale (MFIS)Up to 24 months

    The Modified Fatigue Impact Scale (MFIS) is a revised form of the Fatigue Impact Scale based on items derived from interviews with MS patients concerning how fatigue impacts their lives. This instrument provides an assessment of the effects of fatigue in terms of physical, cognitive, and psychosocial functioning. Participants rate on a 5-point scale, with 0 = 'Never' to 4 = 'Almost always' their agreement with 21 statements. Total score (0-84) and subscales for physical (0-36), cognitive (0-40) and psychosocial functioning (0-8). The 5-item version is scored (0-20). Higher numbers indicate greater fatigue. The MFIS will be performed at the timepoints indicated in protocol.

  • Change from baseline in the assessment of disease as assessed by the Functional Staging for AtaxiaUp to 24 months
  • Overall impression of change as assessed by the patient using the Patient Global Impression of Change (PGI-C) ScaleUp to 24 months

    The Patient Global Impression of Change (PGI-C) reflects a patient's assessment about the efficacy of treatment. PGIC is a 7 point scale depicting a patient's rating of overall improvement. Patients rate their change as "very much improved," "much improved," "minimally improved," "no change," "minimally worse," "much worse," or "very much worse." The PGI-C will be performed at the timepoints indicated in protocol.

  • Overall impression of change assessed by a clinician using the Clinical Global Impression of Change (CGI-C)Up to 24 months

    The Clinical Global Impression of Change (CGI-C) is an assessment to measure change in clinical status (symptoms and functional ability) of the subject's condition from baseline with study drug. CGI-C scores range from 1 (very much improved) through to 7 (very much worse). The CGI-C will be performed at the timepoints indicated in protocol.

  • Area under the concentration-time curve for the dosing interval (AUC0-tau)Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months
  • Area under the concentration-time curve from time 0 to the time of last quantifiable concentration (AUC0-t)Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months
  • Mean maximum observed concentration (Cmax)Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months
  • Mean time of maximum observed concentration (Tmax)Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months
  • Concentration reached immediately before the next dose is administered (Ctrough)Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months